Mission Bio Unveils Industry-First Single-Cell Genotype and Targeted Gene Expression Solution Aimed at Derisking Cancer Drug Clinical Development

• Tapestri Platform's new multi-modal capability enables simultaneous genotype and targeted gene expression profiling at the single-cell level, offering unprecedented insights into tumor heterogeneity and therapeutic response
• Single-cell genotype + targeted gene expression enables high-resolution biomarker identification, and reveals phenotypes and clonal dynamics behind T-cell dysfunction, offering a new level of clarity for cell and gene therapy development
• Mission Bio and collaborators will present new data at European Hematology Association (EHA), held from June 12-15

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Mission Bio, a leader in single-cell multi-omics solutions for precision medicine, today announced the launch of its Single-Cell Genotype and Targeted Gene Expression assay, expanding the capabilities of its Tapestri Platform to become the only commercial solution that delivers simultaneous genotype and targeted gene expression profiling from over 10,000 single cells. The assay, which can be leveraged for Phase 2 or 3 trials to home in on patients most likely to benefit from a cancer therapy, will be unveiled at the European Hematology Association (EHA) meeting in Milan, Italy.

Single-Cell Genotype and Targeted Gene Expression is available through Mission Bio's Pharma Assay Development (PAD) services, where the company's experts provide end-to-end support from custom single-cell assay design and development to data interpretation and assay transfer. The multi-modal assay has three primary applications: helping understand therapeutic resistance, designing next-generation T-cell therapies, and improving patient selection in clinical development.

In particular, the Single-Cell Genotype and Targeted Gene Expression assay addresses critical challenges in oncology drug development, where obtaining the complete picture of tumor heterogeneity and therapeutic resistance mechanisms has been hampered by limitations in existing and disparate methods. By capturing both genomic mutations and targeted gene expression changes in the same cell, the enhanced Tapestri Platform enables pharmaceutical researchers to gain a comprehensive, high-resolution view of cancer cell behavior and treatment response – allowing companies to derisk late-stage development.

“The majority of clinical failures in hematologic cancer drug development occur in Phases II and III, often due to inadequate patient response and lack of predictive biomarkers for patient stratification,” said Brian Kim, CEO of Mission Bio. “We’re directly addressing the urgent need to understand which patients will respond to which drugs at what time, by adding this custom capability to our portfolio of tools. It builds on our continuing mission to offer the tools that drug developers need to understand drug impact at the single-cell level and de-risk late-stage clinical trial failures.”

The new Single-Cell Genotype and Targeted Gene Expression assay also features sample multiplexing capabilities, reducing the cost of single-cell analysis by approximately 60% compared to using multiple technologies to generate similar insights. Tapestri users have previously developed bespoke capabilities to leverage targeted gene expression alongside single-cell genotype and multi-omics, an early demonstration of the value of Mission Bio’s newly integrated approach. Among multiple use cases has been the deconvolution of clonal architecture and tracking resistant subclones under therapeutic pressure in AML. In the future, Mission Bio’s PAD team will enable true multi-omics by adding immunophenotyping to the Single-Cell Genotype and Targeted Gene Expression assay.

Mission Bio and Partners Presenting at EHA

EHA will feature two oral presentations from Mission Bio’s academic partners featuring use of the Tapestri Platform.

Interrogating clonal evolution of NPM1-mutant AML through genetic and genomic approaches

• Dr. Linde Miles, assistant professor at Cincinnati Children's Hospital Medical Center
• Friday, 13 June, 15:45 - 16:15 CEST
• Amber Hall 7+8

Session 3 Keynote lecture: Cell fate and cell plasticity in normal and malignant hematopoiesis

• Dr. Lars Velten, Centre for Genomic Regulation (CRG) Barcelona, Spain
• Saturday, 14 June, 14:00 - 14:20 CEST
• Brown Hall 2

Both Dr. Miles and Dr. Velton will also present at Mission Bio’s EHA evening event, The Power of One: A Night of Multi-Omics Discovery @ EHA 2025, Friday, June 13, 2025 at 7:00 PM.

Late-Breaking Oral Session: INCA33989 is a Novel, First In Class, Mutant Calreticulin-Specific Monoclonal Antibody that Demonstrates Safety and Efficacy in Patients with Essential Thrombocythemia (ET)

• Dr. John Mascarenhas, Icahn School of Medicine at Mount Sinai
• Sunday, June 15, 2025, 09:15 CEST
• Location TBD

Mission Bio will also be presenting six posters at EHA in Poster Hall, beginning on Saturday, 14 June, 18:30 CEST.

• CXCR4 Expression In Aml Blasts And Its Impact On CXCR4 Inhibitor Efficacy During Consolidation Therapy: Results From The Sal Blast Trial
  • Dr. Enise Ceran (Heidelberg, Germany)
• Single Cell Multi-omic Analysis Uncovers Molecular And Clonal Events Associated With Ruxolitinib Treatment Response In Myelofibrosis
  • Dr. Sebastiano Rontauroli (Modena, Italy)
• Characterization Of CD9 As A Novel Marker For Disease-Propagating Stem Cells In Myelofibrosis
  • Dr. Lara Tavernari (Modena, Italy)
• Deciphering Clonal Heterogeneity In Aml Npm1 Through Single-Cell Multi-Omic
  • Dr. Wencke Walter (Munich, Germany)
• Three Patterns Of Molecular Minimal Residual Disease Kinetics In Venetoclax + Azacitidine (V+A) Treated Acute Myeloid Leukemia (AML) Patients
  • Dr. Jiří Mayer (Brno, Czechia)
• High-Throughput And Highly Sensitive Single Cell Genotyping With Simultaneous Chromatin Accessibility Profiling In Myeloid Precursor Conditions
  • Dr. Masanori Motomura (Kyoto, Japan)

For more information about Mission Bio's Targeted Single-Cell Gene Expression assay and Pharma Assay Development services, please visit missionbio.com.

Visit Mission Bio at EHA at booth M.10.

About Mission Bio

Mission Bio is a leading life science company, specializing in the advancement of single-cell DNA and multi-omics analysis. The company's Tapestri Platform is unique in its capabilities, offering an unparalleled level of granularity and precision that is critical for complex research areas such as cancer studies, pharmaceutical development, and advanced cell and gene therapies. Founded in 2014, Mission Bio has secured investment from firms including Mayfield Fund, Novo Growth, Cota Capital, and Agilent Technologies. To learn more about Mission Bio and the Tapestri Platform, please visit missionbio.com.